BioMarin has set up a registry to monitor phenylketonuria (PKU) patients who have received its treatment Kuvan (sapropterin dihydrochloride). The information collected in the database — called the phenylketonuria demographics, outcomes, and safety (PKUDOS) registry (NCT00778206) — will help researchers learn more about the disease in different age groups and study the long-term effectiveness of Kuvan.
What is the PKUDOS registry?
The multicenter PKUDOS registry collects long-term data about patients who are receiving or have received Kuvan. It recruited participants in more than 40 sites in the U.S. The latest update to the government registry’s clinical trial listing, from December 2020, indicated that 1,927 patients had enrolled in the trial. The registry is no longer recruiting and is expected to be completed at the end of January 2021.
What are the goals of PKUDOS?
The goal of the PKUDOS registry is to collect long-term safety and efficacy data on the use of Kuvan in patients of all age groups. The data will show how well Kuvan works at reducing phenylalanine (Phe) levels in patients, and whether it plays a role in the amount of Phe patients are able to consume without having excessive levels in their blood. Finally, the registry will provide useful data on patients who aren’t frequently studied, including pregnant patients and children younger than 4.
What information does PKUDOS collect?
The registry collects data at enrollment and then at regular intervals from quarterly to yearly. This includes medical history, demographics, neurological assessments, adverse events, blood Phe levels, dietary Phe levels, Kuvan dosage, laboratory tests, other medications, genetic screening, blood tyrosine levels, and osteoporosis status.
What do preliminary data show?
As of June 2013, the registry had information on 1,224 patients with 1,189 eligible for analysis. Of those patients, 42% were on continuous Kuvan treatment while 18% had discontinued treatment within the first three months, according to a study published in 2015. The preliminary data showed that for continuous users, Kuvan was able to lower blood Phe levels and increase the tolerable amount of dietary Phe that patients could consume. The data also showed that Kuvan works in a higher number of patients than what the researchers had expected.
New analysis of data through February 2017 found that 1,639 patients had enrolled in the registry with 908 continuously using Kuvan. Patients in this group were able to maintain lower average blood Phe levels while having higher dietary Phe levels than those who had stopped taking the medication (381 patients). The analysis, presented at the National PKU Alliance Conference in 2018, showed that patients tolerated Kuvan fairly well. The most common side effects were digestive issues, nervous system problems, and psychiatric disorders.
Last updated: Jan. 14, 2021
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